Blueprint Medicines and Alexion Partner for Drugs Targeting a Rare Genetic Disease

Rare disease maker Alexion Pharmaceuticals has partnered with Blueprint Medicines to develop drugs for a rare genetic disease.

Blueprint Medicines said that the companies have formed a strategic collaboration to discover, develop and commercialize novel drug candidates for an undisclosed activated kinase target, which causes a rare genetic disease.

Blueprint Medicines will use its kinase-focused drug discovery platform to identify and optimize drug candidates. The company will also conduct all research activities prior to the filing of an investigational new drug (IND) application with the US Food and Drug Administration (FDA). Alexion will be responsible for developing and commercializing any of the drug candidates resulting from the collaboration.

“Our kinase-focused platform, which integrates a novel target discovery engine and a proprietary compound library, enables us to craft highly selective kinase drugs for genomic drivers of disease across many therapeutic areas. Alexion is the ideal partner for our rare genetic disease program with their successful track record developing and commercializing therapies for severe and life-threatening disorders,” said Jeffrey Albers, Chief Executive Officer of Blueprint Medicines. “Working with Alexion on this target will allow the team at Blueprint Medicines to focus on our primary strategic area of oncology, while we leverage our platform in additional therapeutic areas.”

In addition to an upfront fee of $15 million, Alexion will reimburse Blueprint Medicines for all research expenses. Additionally, Blueprint Medicines is eligible to receive more than $250 million in milestone payments and will be eligible for royalty payments for any commercialized products.

This is Blueprint Medicines’ first strategic collaboration since its formation in 2011, when Third Rock Ventures and Fidelity Biosciences provided $40 million in Series A. Since then, the company has brought in $115 million in funding. Blueprint Medicines has focused on oncology products to date, with two products expected to enter the clinic by the end of 2015.

“Blueprint Medicines’ unique discovery platform enables it to create drug candidates for extremely challenging kinase targets. Even in these early stages, Blueprint Medicines’ compounds show impressive selectivity toward the mutant kinase, thereby sparing other kinases and delivering drug to the specified target,” said Martin Mackay, PhD, Executive Vice President, Global Head of Research and Development at Alexion. “We look forward to partnering with the talented Blueprint Medicines’ team to advance a highly innovative therapy for patients suffering from a devastating rare genetic disease.”

Source: Blueprint Medicines

Last updated: 3/3/15; 2:40pm EST

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